GIST Specialist for San Marino, CA
For most localised gastrointestinal stromal tumors the standard pathway is the right one, and a clinical trial adds nothing. For resistant and wild type disease the picture is different. Joshua Ellenhorn, MD, FACS will tell you which applies to you.
- Honest assessment of whether a trial is relevant to your case
- Clinical Professor of Surgery, Cedars-Sinai Medical Center
- Practises within a comprehensive cancer institute
- Thirty to thirty five minutes via the 110 and 101
A Trial Is Not What Happens When Nothing Is Left
Most people first hear about clinical trials in the context of desperation, and that framing does real harm. It leads patients to reject a trial early on as premature, then consider one much later when they are too unwell to be eligible for anything.
In practice trials exist across the whole spectrum. Some test whether an established treatment can be improved, shortened, or better targeted. Some compare two approaches that are both already accepted. Some ask whether a drug that works in advanced disease also helps earlier. None of that is a substitute for treatment that works.
For gastrointestinal stromal tumors specifically, the honest picture is this. If you have a localised, resectable tumor, the standard pathway of complete surgical removal with adjuvant therapy where the risk grading warrants it is genuinely good treatment, and there is usually no reason to look further. Saying so is more useful than implying every patient should be seeking something experimental.
Where trials matter considerably more is in two situations. Wild type and SDH-deficient tumors respond poorly to the existing drugs, so the established sequence offers less. And disease that has progressed through the recognised lines of therapy has, by definition, exhausted what is standard. In both cases a trial may represent the most promising option available rather than a fallback.
What the Phases Actually Mean
Understanding the structure makes it much easier to judge whether a particular trial is worth considering.
| Phase | What it is testing | What it means for you |
|---|---|---|
| Phase one | Safety and appropriate dose, usually in a small group across several cancer types | Earliest stage. Benefit is uncertain by design, and these are generally considered when standard options are exhausted |
| Phase two | Whether the treatment shows activity in a specific disease | More focused. Evidence of possible benefit exists, though it has not been compared against standard care |
| Phase three | The new approach compared against current standard treatment, with randomisation | Largest and most rigorous. You receive either the new approach or established care, not nothing |
| Phase four | Longer term outcomes after a treatment is already approved | Usually involves treatment you would be receiving anyway |
| Randomisation | Which arm you are assigned to is decided by chance rather than choice | The mechanism that makes comparison meaningful. It also means you cannot select your arm |
| Eligibility criteria | Specific requirements about tumor type, prior treatments, organ function | Frequently the practical barrier. Prior lines of therapy in particular can exclude you from some trials and qualify you for others |
Placebos are rarely used alone in cancer trials. Where they appear it is usually alongside standard treatment rather than instead of it, and the design is set out in the consent documents.
The Subtypes Where Standard Drugs Fall Short
Nearly all gastrointestinal stromal tumors are driven by a mutation in KIT or PDGFRA, and imatinib was designed to block exactly that signal. In those tumors it works remarkably well, which is why the standard pathway is genuinely strong.
Some tumors do not fit that description. Wild type GIST, where no KIT or PDGFRA mutation is found, and SDH-deficient GIST, defined by loss of the SDHB protein, both respond poorly to imatinib. These occur disproportionately in younger patients. For them the established drug sequence offers considerably less, and a trial is a more reasonable early consideration rather than a late one.
The other group is patients whose disease has progressed through the recognised lines: imatinib, then sunitinib, then regorafenib, then ripretinib. Reaching the end of that sequence is precisely when a trial becomes the leading option, and being referred at that point rather than several months later is worth pressing for.
Knowing which group you are in requires mutation testing. If it was never done, that is the first thing to address, because it determines both which drugs will work and which trials you might be eligible for.
The Surgery, Which Remains Central
Recordings of real GIST resections narrated by the surgeon who performed them. Whatever else is being considered, complete removal of a localised tumor is what cures this disease.
Laparoscopic Resection of a Gastric GIST
A gastric GIST removed through small incisions with the capsule kept intact.
Gastric GIST Resection: A Simplified Approach
A refined approach that shortens operating time and recovery.
Robotic Resection of a Duodenal GIST
Duodenal GIST removed robotically with the pancreas and bile duct preserved.
Was Mutation Testing Done?
It determines which drugs can work and which trials you may be eligible for. Wild type and SDH-deficient tumors in particular change the calculation entirely, and both are identified by testing rather than by appearance.
Certifications and Appointments
Two American Board of Surgery certifications, a Cedars-Sinai teaching appointment, and membership of the Society of Surgical Oncology and the American Society of Clinical Oncology.
- American Board of Surgery, certified in General Surgery
- American Board of Surgery, certified in Colorectal Surgery
- California State Medical License
- Florida State Medical License
Joshua Ellenhorn, MD, FACS
Dr. Ellenhorn is a surgical oncologist and Clinical Professor of Surgery at Cedars-Sinai Medical Center, and a collaborative member of the Samuel Oschin Comprehensive Cancer Institute.
Trial enrolment is led by medical oncology rather than by surgeons, and this page is not an offer of one. What a surgical opinion contributes is the other half of the question: whether an operation still has a role alongside whatever drug strategy is being pursued. Patients considering a trial for advanced disease are sometimes also candidates for resecting a single progressing deposit, and those two possibilities are frequently assessed in isolation from each other when they should be weighed together.
He is certified by the American Board of Surgery in General Surgery and in Colorectal Surgery, a Fellow of the American College of Surgeons, and a member of the Society of Surgical Oncology and the American Society of Clinical Oncology. He practises with the Surgery Group of Los Angeles.
Questions Worth Asking
Take these to the oncologist managing your treatment. The first two determine whether the rest are even relevant.
- What is my mutation subtype?
- Am I wild type or SDH-deficient?
- Which lines of therapy have I already had?
- Are there trials appropriate for my subtype or resistance pattern?
- What phase is the trial, and what is it comparing against?
- What would be required of me practically, including travel and visits?
- Would joining a trial affect my eligibility for later treatment?
- Does surgery still have a role alongside whatever drug strategy we choose?
What Patients Say
Reviews left by patients treated by Dr. Ellenhorn at the Surgery Group of Los Angeles.
★★★★★Very kind stuff, great service. Dr Joshua Ellenhorn is a very calm doctor who takes time with his patient.
Anita Lukacevic
★★★★★I was very pleased with the office staff, they were always very pleasant and helpful. I was very impressed with Dr. Ellenhorn as well. He was very friendly and addressed all my concerns regarding my surgery and aftercare and follow up.
Terry Jackson
From San Marino
The 110 south to the 101, or the 134 west to the 101, then Highland down and west along 3rd Street. Thirty to thirty five minutes in ordinary conditions, longer through the Cahuenga Pass in the evening. From the Huntington Drive end, the 110 is usually the cleaner line.
The office is in the medical plaza attached to Cedars-Sinai Medical Center, within the Samuel Oschin Comprehensive Cancer Institute. For patients weighing trial participation, being assessed inside a comprehensive cancer centre means the surgical and medical questions are considered in the same place rather than sequentially across institutions.
Patients travelling from outside the region, including Arizona and Nevada, can have imaging and pathology reviewed remotely.
8635 W 3rd St, Suite 880W
Los Angeles, CA 90048
Monday to Saturday, 11:00am to 8:00pm
Closed Sunday
Frequently Asked Questions
Are clinical trials a last resort?
Do I need a trial if my GIST is localised and resectable?
What do the phases mean?
If I join a trial, could I be given a placebo instead of treatment?
Which GIST patients most often benefit from a trial?
Where do I look for trials?
Would I have to travel?
How far is your office from San Marino?
Trials Matter Most for Wild Type and Resistant Disease.
If your tumor is localised and resectable, standard treatment is genuinely good. If it is wild type, SDH-deficient, or has progressed through the established drugs, that is when to look seriously.
